Methods for identifying and treating hemoglobinopathies

Abstract

The invention relates to method of treating or inhibiting progression of hemoglobinopathy in a subject in need thereof comprising inhibiting interaction between LRF-BTB and CHD protein-LBD.

Description

INCORPORATION BY REFERENCE[0001]This application claims benefit of and priority to U.S. provisional patent application 62 / 272,853 filed Dec. 30, 2015, incorporated herein by reference.[0002]All documents cited or referenced herein (“herein cited documents”), and all documents cited or referenced in herein cited documents, together with any manufacturer's instructions, descriptions, product specifications, and product sheets for any products mentioned herein or in any document incorporated by reference herein, are hereby incorporated herein by reference, and may be employed in the practice of the invention. More specifically, all referenced documents are incorporated by reference to the same extent as if each individual document was specifically and individually indicated to be incorporated by reference.[0003]Mention is made of Abstract No. 80611 and Presentation “The LRF / ZBTB7A Transcription Factor is a BCL11A-Independent Repressor of Fetal Hemoglobin” presented at the American Soci...

AI Technical Summary

Benefits of technology

The patent is about a process called high-throughput screening (HTS) that uses robotics and sensitive detectors to quickly test millions of compounds for their effects on biological pathways. This process can identify active compounds that could be used to treat disorders or conditions. The invention also provides targets for developing specific drugs and a way to test the safety of potential therapies. The method is efficient, automated, and can handle large numbers of compounds. Overall, the patent presents a powerful tool for identifying and developing new treatments for biological disorders.

Problems solved by technology

However, mechanisms that repress HbF in adults remain unclear.

Nuclear factors that regulate globin switching have been identified, but how they function cooperatively or independently in fetal globin repression is not fully understood.

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